The FDA’s decision was not unexpected, as the proprietary extract formulation — developed by the British biotechnology firm GW Pharmaceuticals — had previously demonstrated safety and clinical efficacy at reducing seizure frequency in several placebo-controlled trials. Epidiolex had previously received Fast Track Designation and Orphan Drug Status from the FDA. It is the fourth marijuana-based medicine to receive US FDA approval — joining dronabinol (aka Marinol), nabilone (aka Cesamet), and liquid synthetic THC (aka Syndros). However, Epidiolex is the first FDA-approved medicine containing plant-derived, non-synthetic cannabinoids.

Commenting on the agency’s decision, NORML Deputy Director Paul Armentano said: “The FDA’s approval of this plant-derived medicine provides an additional option to patients seeking the therapeutic benefits of cannabis. However, it remains to be seen whether physicians will be comfortable prescribing this new agent to those patients who may benefit from it, and whether it will be priced in a range that patients may afford.” According to the New York Times, analysts expect Epidiolex to cost $2,500 to $5,000 a month.

He added: “We anticipated that Epidiolex will be the first of many potential FDA-approved medicines based on the cannabis plant. Nonetheless, these alternatives should not be regulated as options to replace the use and regulation of herbal cannabis — a product that humans have used safely and effectively as a medicine for thousands of years and is approved today by statute in 30 states.”

Federal agencies have 90 days to determine the scheduling of Epidiolex. The new drug is anticipated to become available to patients later this fall. In clinical trials, patients administered Epidiolex, on average, obtained a 40 percent reduction in seizure frequency.

Lennox-Gaustaut syndrome is estimated to account for between one and four percent of all cases of childhood epilepsy. Dravet syndrome is estimated to effect about 1 in 40,000 people.

Despite today’s approval, the FDA acknowledged in a statement that the cannabidinoid CBD still remains classified at this time as a schedule I controlled substance, and that the agency is “prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims.” FDA Commissioner Scott Gottlieb further added: “This is the approval of one specific CBD medication for a specific use. … [T]his is not an approval of marijuana or all of its components.”

The administration of cannabis oil extracts high in cannabidiol reduces seizure frequency in children with intractable epilepsy, according to clinical data published online ahead of print in the journal Seizure.

Israeli researchers retrospectively evaluated the effects of CBD oil in a multicenter cohort of 74 patients with intractable epilepsy. Participants in the trial were resistant to conventional epilepsy treatment and were treated with CBD extracts for a period of at least three months. Extracts in the study were provided by a pair of Israeli-licensed growers and were standardized to possess a CBD to THC ratio of 20 to 1.

Investigators reported: “CBD treatment yielded a significant positive effect on seizure load. Most of the children (89 percent) reported reduction in seizure frequency. … In addition, we observed improvement in behavior and alertness, language, communication, motor skills and sleep.”

They concluded, “The results of this multicenter study on CBD treatment for intractable epilepsy in a population of children and adolescents are highly promising. Further prospective, well-designed clinical trials using enriched CBD medical cannabis are warranted.”

Previously published retrospective studies and surveys, such as those here and here and here, have also reported CBD administration to be efficacious in reducing seizure frequency.

In 2013, the United States Food and Drug Administration granted orphan drug status to imported, pharmaceutically standardized CBD (aka Epidiolex) extracts for use in experimental pediatric treatment. Open-label safety trial data published online in December 2015 in the journal Lancet Neurology reported a median reduction in seizures in adolescent patient treated with Epidiolex that approached 40 percent. Authors concluded, “Our findings suggest that cannabidiol might reduce seizure frequency and might have an adequate safety profile in children and young adults with highly treatment-resistant epilepsy.”

An abstract of the study, “CBD-enriched medical cannabis for intractable pediatric epilepsy: The current Israeli experience,” appears online here.

The administration of oral cannabis extracts is associated with the mitigation of seizures in adolescents with epilepsy, according to clinical data published this month in the journal Epilepsy & Behavior.

Researchers from the Colorado Children’s Hospital in Denver performed a retrospective chart review of 75 children provided cannabis extracts. Authors reported that 57 percent of subjects showed some level of improvement in seizure control while 33 percent reported a greater than 50 percent reduction in seizure frequency.

Researchers also reported “improved behavior/alertness” in one-third of subjects and improved motor skills in ten percent of treated patients. Adverse events were reported in 44 percent of subjects, 13 percent of which reported increased seizure activity. Overall, however, authors concluded that the extracts were “well tolerated by children.”

Separate clinical trial results publicized last week at the 67th Annual Meeting of the American Academy of Neurology reported that the administration of a proprietary form of CBD (cannabidiol) extracts decreased seizure frequency by 54 percent over a 12-week period in children with treatment-resistant epilepsy.

Survey data compiled by Stanford University in 2013 reported that the administration of cannabidiol-enriched cannabis decreased seizures in 16 of 19 patients with pediatric epilepsy.

Last February, the Epilepsy Foundation of America enacted a resolution in support of the “rights of patients and families living with seizures and epilepsy to access physician directed care, including medical marijuana.”

An abstract of the study, “Parental reporting of response to oral cannabis extracts for treatment of refractory epilepsy,” appears online here.

State Public Health Department officials have recommended over $7 million dollars in grant funding to pay for a series of state-sponsored clinical trials to assess the safety and efficacy of cannabis and cannabinoids.

The proposed studies include a pair of clinical trials to evaluate the use of cannabidiol (CBD), a nonpsychotropic plant cannabinoid, for patients with pediatric epilepsy. Two additional trials will assess the use of cannabis for patients suffering from post-traumatic stress. Other studies will assess the efficacy of either cannabis or CBD in the treatment of Parkinson’s disease, brain tumors, ulcerative colitis, and pain management. (More specific summaries of all eight proposed studies are available online here.)

Grant funding for the proposed studies requires final approval by the state Board of Health in December.

Following funding approval, researchers will still be required to gain additional federal approval in order to obtain access to research-grade cannabis or CBD.

The state of California previously sponsored a similar series of clinical trials assessing the safety and efficacy of marijuana. Those studies evaluated the use of whole-plant cannabis in patients with neuropathy, multiple sclerosis, and autoimmune deficiencies. A summary of those trials, published in 2012, concluded, “Based on evidence currently available the Schedule I classification is not tenable; it is not accurate that cannabis has no medical value, or that information on safety is lacking.”

At a public signing ceremony today, Utah’s Republican Gov. Gary Herbert will approve House Bill 105 — aka “Charlee’s Law.” The law, and others like it, ostensibly allows children with treatment-resistant pediatric epilepsy access to extracts of the marijuana plant high in the cannabinoid cannabidiol (CBD).

While some media outlets are reporting that the passage of these measures are akin to ‘approving medical marijuana,’ such claims are far from accurate.

Specifically, Utah’s HB 105 — which takes effect on July 1 — is largely unworkable. As written, the law only exempts from state prosecution those with “intractable epilepsy” who possess a cannabis extract that contains more than 15 percent CBD and no more than 0.3 percent THC. Patients must receive a written recommendation from a Utah board certified neurologist and be registered with the state Department of Health before seeking such extracts, which for the time being may only be procured from outside of the state. The extracts must be produced in a lab that possess a state-license to manufacture such products.

While this language may appear to allow Utah patients to procure CBD products in neighboring medical cannabis states like Colorado, the likelihood of this scenario is highly doubtful. Colorado’s medical marijuana law only allows those who are state residents and who possess a state-issued patient identification card to legally purchase such products. In other words, Utah parents would have to violate Colorado law to obtain high-CBD extracts (which are likely to only be available from a medical dispensary, not a retail cannabis market). Colorado medical marijuana dispensaries would also be in violation of not just the letter of the law, but also the spirit of the law by providing a product they know is intended to be transported across state lines — a clear violation of the guidelines put forward in the August 2013 Department of Justice memo which call for “preventing the diversion of marijuana from states where it is legal in some form to other states.”

Utah’s forthcoming law also calls on the state Department of Agriculture “to grow or cultivate industrial hemp for the purpose of
agricultural or academic research,” ostensibly for the purpose of one-day producing high-CBD cannabis medicines. However, it remains to be seen whether such industrial crops can yield therapeutically effective CBD extracts or whether federal lawmakers would even allow such a state-sponsored research project to move forward.

In Alabama, members of the House and Senate unanimously approved CBD-specific legislation, Senate Bill 174 aka “Carley’s Laws,” late last week. Republican Gov. Robert Bently has announced his intent to sign the measure into law.

However, like the Utah law, Alabama’s forthcoming law will also be largely unworkable for those who seek to benefit from it. The measure appropriates $1 million dollars for University of Alabama-sponsored research in CBD extracts. Whether such research will actually take place is another story. Because CBD is, like the cannabis plant itself, classified under federal law as a schedule I controlled substance, multiple federal agencies — including the FDA, DEA, NIDA (US National Institute of Drug Abuse), and PHS (Public Health Service) must all sign off on any clinical investigation of the drug — a process that typically takes several years and often ends with federal regulators rejecting the protocol outright. Yet, under “Carley’s Law,” patients may only legally access CBD under if it is “prescribed” during the course of such a federally approved clinical trial.

Nevertheless, despite these obvious limitations in implementation, lawmakers in various other states — including Florida, Kentucky, Minnesota, South Carolina, and Wisconsin — are considering passing similar measures. (A similar Georgia measure died when lawmakers adjourned late last week.) While the passage of these measures may pose symbolic victories for legislators, they fail to provide tangible benefits to the constituents that they are intended to serve.